Spark Therapeutics
Company type | Subsidiary of Hoffmann-La Roche |
---|---|
Industry | Biotechnology Pharmaceutical |
Founded | 2013 |
Founders | Katherine A. High Jeffrey Marrazzo Jean Bennett J Fraser Wright Beverly Davidson Jennifer Wellman |
Headquarters | Philadelphia, Pennsylvania |
Revenue | $64 million (2018) |
-$78 million (2018) | |
Total assets | $814 million (2018) |
Total equity | $496 million (2018) |
Number of employees | 368 (2019) |
Parent | Hoffmann-La Roche |
Website | sparktx |
Footnotes / references [1] |
Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases.[1] It is a subsidiary of Hoffmann-La Roche.
History
The company was founded in 2013 by Katherine A. High, Jeffrey Marrazzo, and Steven Altschuler[2] in an effort to commercially develop treatments against haemophilia that High was working on at Children's Hospital of Philadelphia.[3]
In January 2015, the company became a public company, trading under the ticker $ONCE via a $161 million initial public offering [4] led by Chief Legal Officer Joseph La Barge.[5]
In December 2017, the U.S. Food and Drug Administration approved LUXTURNATM (voretigene neparvovec-rzyl) for the treatment of patients with viable retinal cells and confirmed biallelic RPE65 mutation-associated retinal dystrophy, a genetic blinding condition caused by mutations in the RPE65 gene.[1]The company is currently developing several gene therapies to target a suite of diseases, including Haemophilia A and B, and several central nervous system diseases.
In December 2019, the company was acquired by Hoffmann-La Roche for $4.3 billion. [6][7]It now continues to operate as an independent subsidiary. [8] Since the acquisition by Swiss pharma Roche, several key founding executives have departed, including scientist and co-founder Katherine High in February 2020 [9], Chief Business/Legal Officer Joseph La Barge in December 2021, and co-founder and Chief Executive Officer Jeffrey Marrazzo in April 2022 [10]
On February 23rd, 2022, Marrazzo named big-Pharma veteran Ron Philip as his successor. Mr. Philip currently leads the organization[11].
Products and pipeline
The company has 3 gene therapy product candidates in clinical development: (i) SPK-8011, a candidate in the SPK-FVIII program for hemophilia A; (ii) SPK-8016, a product candidate for the hemophilia A inhibitor market; and (iii) SPK-7001, targeting choroideremia, or CHM. SPK-9001, a lead product candidate in the SPK-FIX program for hemophilia B, is being developed in partnership with Pfizer.[1]
Voretigene neparvovec
Voretigene neparvovec, marketed under the tradename Luxturna, is a gene therapy approved by the Food and Drug Administration for treatment of Leber's congenital amaurosis, a rare genetic eye disease.[12]
Fidanacogene elaparvovec
Fidanacogene elaparvovec, previously known by its study ID number SPK-9001,[13] is an experimental drug under investigation for treatment of hemophilia B in partnership with Pfizer. Fidanacogene elaparvovec is an adeno-associated viral vector which is designed to transfer a working copy of the Factor IX gene into the livers of patients who carry non-functioning copies.[14] In July 2018, fidanacogene elaparvovec entered late stage clinical trials.[15]
SPK-8011
SPK-8011 is an experimental drug under investigation for treatment of Haemophilia A. It is entering phase III clinical trials in the United States. The therapy transfers a working copy of the Factor VIII gene into patients who lack one. In Phase II clinical trials, 2 of 7 patients receiving the highest dose of the drug suffered immune responses. One patient had to be hospitalized. The reactions against the treatment were seen as a set-back, though Spark suggested that the responses could be controlled with steroids, and promised to move forward with Phase III testing.[16][17]
SPK-7001
SPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness.[18]
SPK-3006
SPK-3006 is an experimental drug under investigation for treatment of Pompe disease, a genetic disorder that leads to failure to correctly metabolize glycogen.[18]
SPK-1001
SPK-1001 is an experimental drug under investigation for treatment of Batten disease, a fatal genetic, nervous system disorder.[18]
References
- ^ a b c d "Spark Therapeutics, Inc. 2018 Form 10-K Annual Report". U.S. Securities and Exchange Commission.
- ^ Higgins, Robert F., and Tina Liu. "Spark Therapeutics: Pioneering Gene Therapy." Harvard Business School Case 818-059, January 2018.
- ^ Crow, David (19 October 2017). "Gene therapy helped these children see. Can it transform medicine?". Financial Times.
- ^ https://www.fiercebiotech.com/r-d/spark-nails-a-161m-ipo-to-fund-its-breakthrough-gene-therapy
- ^ George, John (January 30, 2015). "Shake Shack wasn't the day's only gonzo IPO. And this one, for Spark Therapeutics, raised more cash". American City Business Journals.
- ^ "Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics". American City Business Journals. 17 December 2019.
- ^ "Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy" (Press release). Hoffmann-La Roche. 17 December 2019.
- ^ "Roche concludes acquisition of Spark Therapeutics, Inc. To strengthen presence in gene therapy".
- ^ "Spark Co-Founder Katherine High Departs Company Ahead of Merger with Roche". BioSpace. Retrieved 2020-03-03.
- ^ https://sparktx.com/press_releases/spark-therapeutics-announces-departure-of-ceo-and-founder-jeff-marrazzo-coo-ron-philip-named-as-successor/
- ^ https://sparktx.com/press_releases/spark-therapeutics-announces-departure-of-ceo-and-founder-jeff-marrazzo-coo-ron-philip-named-as-successor/
- ^ "FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss" (Press release). Food and Drug Administration. 19 December 2017.
- ^ "Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy" (Press release). Pfizer. 16 July 2018.
- ^ Lindsey, George (1 December 2016). "Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression". Blood. 128 (22): 3. doi:10.1182/blood.V128.22.3.3.
- ^ George, John (16 July 2018). "Pfizer begins late-stage testing of Spark's hemophilia B gene therapy". American City Business Journals.
- ^ Garde, Damian (7 August 2018). "Spark's gene therapy data answer some burning questions — and raise a few more". Stat.
- ^ Tirrell, Meg (7 August 2018). "Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors". CNBC.
- ^ a b c Morrison, Chris (5 March 2019). "Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal". Nature Biotechnology.
External links
- Biotechnology companies of the United States
- Pharmaceutical companies of the United States
- Biopharmaceutical companies
- Haemophilia
- Pharmaceutical companies established in 2013
- Biotechnology companies established in 2013
- 2013 establishments in Pennsylvania
- Companies based in Philadelphia
- Health care companies based in Pennsylvania
- Virotherapy
- Companies formerly listed on the Nasdaq
- 2015 initial public offerings
- 2019 mergers and acquisitions
- Roche
- American subsidiaries of foreign companies